Ignite Creation Date:
2025-12-24 @ 11:51 PM
Ignite Modification Date:
2026-02-25 @ 4:37 PM
Study NCT ID:
NCT01812551
Status:
COMPLETED
Last Update Posted:
2014-03-06
First Post:
2013-03-14
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Treatment of Low Bone Density in Cystic Fibrosis.
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D010024', 'term': 'Osteoporosis'}, {'id': 'D003550', 'term': 'Cystic Fibrosis'}, {'id': 'D050723', 'term': 'Fractures, Bone'}], 'ancestors': [{'id': 'D001851', 'term': 'Bone Diseases, Metabolic'}, {'id': 'D001847', 'term': 'Bone Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D010182', 'term': 'Pancreatic Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D008171', 'term': 'Lung Diseases'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D007232', 'term': 'Infant, Newborn, Diseases'}, {'id': 'D014947', 'term': 'Wounds and Injuries'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D019386', 'term': 'Alendronate'}], 'ancestors': [{'id': 'D004164', 'term': 'Diphosphonates'}, {'id': 'D063065', 'term': 'Organophosphonates'}, {'id': 'D009943', 'term': 'Organophosphorus Compounds'}, {'id': 'D009930', 'term': 'Organic Chemicals'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 171}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2002-10'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2014-03', 'completionDateStruct': {'date': '2007-07', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2014-03-05', 'studyFirstSubmitDate': '2013-03-14', 'studyFirstSubmitQcDate': '2013-03-15', 'lastUpdatePostDateStruct': {'date': '2014-03-06', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2013-03-18', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2006-07', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Bone mineral density increase at lumbar spine.', 'timeFrame': 'up to 24 months', 'description': 'Bone mineral density evaluated by DXA. Bone mineral apparent density calculated to correct for bone size (growing subjects). Z-score calculated.\n\nMeasurements: Phase 1 (171 subjects): Baseline, 6 months, 12 months. Phase 2 (128 subjects, randomized to 2 arms: placebo or alendronate): 18 months, 24 months.'}], 'secondaryOutcomes': [{'measure': 'Changes in bone turnover markers.', 'timeFrame': 'baseline and up to 24 months', 'description': 'Bone turnover markers: (serum) osteocalcin (OC), bone specific alkaline phosphatase (BSAP), C-terminal telopeptide of procollagen 1 (CTx); (urine) terminal telopeptide of procollagen 1 (NTx).'}, {'measure': 'Fracture rate.', 'timeFrame': 'at 12th and 24th month', 'description': 'Appendicular fractures were evaluated at baseline (previous fractures) and throughout the 2 years of study (incident fractures) with X-rays.\n\nVertebral fractures were evaluated at the end of Phase 1 (12th month) and at the end of Phase 2 (24th month) with lateral thoracic and lumbar spine X-rays.'}, {'measure': 'Adverse effects of alendronate.', 'timeFrame': 'continuously throughout Phase 2 (2nd year of study)', 'description': 'Evaluated on the basis of lab tests (calcemia, calciuria, blood cell count, liver and kidney function), FEV1 changes, and other signs/symptoms (e.g. pain, fever, etc.)'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['cystic fibrosis', 'children', 'adolescents', 'low bone mass', 'osteoporosis', 'alendronate', 'fractures'], 'conditions': ['Osteoporosis', 'Cystic Fibrosis']}, 'referencesModule': {'references': [{'pmid': '8346485', 'type': 'BACKGROUND', 'citation': 'Grey AB, Ames RW, Matthews RD, Reid IR. Bone mineral density and body composition in adult patients with cystic fibrosis. Thorax. 1993 Jun;48(6):589-93. doi: 10.1136/thx.48.6.589.'}, {'pmid': '8551418', 'type': 'BACKGROUND', 'citation': 'Henderson RC, Madsen CD. Bone density in children and adolescents with cystic fibrosis. J Pediatr. 1996 Jan;128(1):28-34. doi: 10.1016/s0022-3476(96)70424-9.'}, {'pmid': '8545201', 'type': 'BACKGROUND', 'citation': 'Bhudhikanok GS, Lim J, Marcus R, Harkins A, Moss RB, Bachrach LK. Correlates of osteopenia in patients with cystic fibrosis. Pediatrics. 1996 Jan;97(1):103-11.'}, {'pmid': '9078542', 'type': 'BACKGROUND', 'citation': 'Baroncelli GI, De Luca F, Magazzu G, Arrigo T, Sferlazzas C, Catena C, Bertelloni S, Saggese G. Bone demineralization in cystic fibrosis: evidence of imbalance between bone formation and degradation. Pediatr Res. 1997 Mar;41(3):397-403. doi: 10.1203/00006450-199703000-00016.'}, {'pmid': '9569551', 'type': 'BACKGROUND', 'citation': 'Humphries IR, Allen JR, Waters DL, Howman-Giles R, Gaskin KJ. Volumetric bone mineral density in children with cystic fibrosis. Appl Radiat Isot. 1998 May-Jun;49(5-6):593-5. doi: 10.1016/s0969-8043(97)00203-0. No abstract available.'}, {'pmid': '9454526', 'type': 'BACKGROUND', 'citation': 'Aris RM, Renner JB, Winders AD, Buell HE, Riggs DB, Lester GE, Ontjes DA. Increased rate of fractures and severe kyphosis: sequelae of living into adulthood with cystic fibrosis. Ann Intern Med. 1998 Feb 1;128(3):186-93. doi: 10.7326/0003-4819-128-3-199802010-00004.'}, {'pmid': '9672505', 'type': 'BACKGROUND', 'citation': 'Bhudhikanok GS, Wang MC, Marcus R, Harkins A, Moss RB, Bachrach LK. Bone acquisition and loss in children and adults with cystic fibrosis: a longitudinal study. J Pediatr. 1998 Jul;133(1):18-27. doi: 10.1016/s0022-3476(98)70172-6.'}, {'pmid': '10088930', 'type': 'BACKGROUND', 'citation': 'Henderson RC, Madsen CD. Bone mineral content and body composition in children and young adults with cystic fibrosis. Pediatr Pulmonol. 1999 Feb;27(2):80-4. doi: 10.1002/(sici)1099-0496(199902)27:23.0.co;2-j.'}, {'pmid': '11014345', 'type': 'BACKGROUND', 'citation': 'Bianchi ML, Cimaz R, Bardare M, Zulian F, Lepore L, Boncompagni A, Galbiati E, Corona F, Luisetto G, Giuntini D, Picco P, Brandi ML, Falcini F. Efficacy and safety of alendronate for the treatment of osteoporosis in diffuse connective tissue diseases in children: a prospective multicenter study. Arthritis Rheum. 2000 Sep;43(9):1960-6. doi: 10.1002/1529-0131(200009)43:93.0.CO;2-J.'}, {'pmid': '9279333', 'type': 'BACKGROUND', 'citation': 'Brumsen C, Hamdy NA, Papapoulos SE. Long-term effects of bisphosphonates on the growing skeleton. Studies of young patients with severe osteoporosis. Medicine (Baltimore). 1997 Jul;76(4):266-83. doi: 10.1097/00005792-199707000-00005.'}, {'pmid': '9753709', 'type': 'BACKGROUND', 'citation': 'Glorieux FH, Bishop NJ, Plotkin H, Chabot G, Lanoue G, Travers R. Cyclic administration of pamidronate in children with severe osteogenesis imperfecta. N Engl J Med. 1998 Oct 1;339(14):947-52. doi: 10.1056/NEJM199810013391402.'}, {'pmid': '24429202', 'type': 'DERIVED', 'citation': 'Bianchi ML, Colombo C, Assael BM, Dubini A, Lombardo M, Quattrucci S, Bella S, Collura M, Messore B, Raia V, Poli F, Bini R, Albanese CV, De Rose V, Costantini D, Romano G, Pustorino E, Magazzu G, Bertasi S, Lucidi V, Traverso G, Coruzzo A, Grzejdziak AD. Treatment of low bone density in young people with cystic fibrosis: a multicentre, prospective, open-label observational study of calcium and calcifediol followed by a randomised placebo-controlled trial of alendronate. Lancet Respir Med. 2013 Jul;1(5):377-85. doi: 10.1016/S2213-2600(13)70064-X. Epub 2013 Jun 2.'}], 'seeAlsoLinks': [{'url': 'http://www.lios.it/research/telethon/Bianchi_CF-protocol-2000.pdf', 'label': 'Study protocol'}, {'url': 'http://www.lios.it/research/telethon/Bianchi_CF-protocol-2000-labtests.pdf', 'label': 'Lab test information'}]}, 'descriptionModule': {'briefSummary': 'Cystic fibrosis (CF) -- an autosomal recessive genetic disease affecting about 60,000 individuals worldwide, including about 3,800 in Italy -- is often associated with low bone mineral mass. The current aggressive therapies have ensured a much longer survival of CF patients but this has led to a higher frequency of osteoporosis and bone fractures, a serious problem which not only affects quality of life, but also hinders further therapeutic measures.\n\nThe aim of this study, conducted on a large group of children, adolescents and young adults with CF, has been the evaluation of bone mass changes after 1 year of a simple treatment with RDA-adjusted dietary calcium plus 25-OH vitamin D supplementation, and the feasibility and efficacy of alendronate treatment (for another year) in patients not responding to calcium + 25-OH vitamin D alone.', 'detailedDescription': "The study included 2 phases.\n\nPhase 1 (1-year open-label observational study): following baseline evaluation, bone mass changes have been studied with a simple therapy of adequate calcium intake and 25-OH vitamin D supplements in all eligible subjects (N=171).\n\nPhase 2 (1-year double-blind, randomized, placebo-controlled, parallel group study): the 128 subjects showing an insufficient response to calcium + 25-OH vitamin D alone (bone mass increase \\<5%) at the end of Phase 1, were randomized into 2 groups and assigned to alendronate treatment (N=65) or placebo (N=63) (in addition to calcium and 25-OH vitamin D as during Phase 1).\n\nThe study has been carried out by the Coordinator's Institution (Istituto Auxologico Italiano)in collaboration with most Regional Reference Centers for CF in Italy."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '30 Years', 'minimumAge': '5 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* age 2-30 years\n* clinically stable condition\n* regular menses in females\n* low Bone Mineral Apparent Density for age (defined as BMAD Z-score ≤-2.0 if age ≤18 years or ≤-2.5 if age \\>18 years).\n\nExclusion Criteria:\n\n* two or more episodes of hypercalcemia and/or hypercalciuria\n* contraindications to 25-OH vitamin D or alendronate treatment\n* recent transplantation\n* other diseases or medications (glucocorticoids excepted) associated with bone loss.'}, 'identificationModule': {'nctId': 'NCT01812551', 'acronym': 'OSCYF', 'briefTitle': 'Treatment of Low Bone Density in Cystic Fibrosis.', 'organization': {'class': 'OTHER', 'fullName': 'Istituto Auxologico Italiano'}, 'officialTitle': 'Osteoporosis in Cystic Fibrosis: Study of Bone Mass and Bone Metabolism, and Prospective Randomized Therapeutic Trial.', 'orgStudyIdInfo': {'id': '02A001'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'ACTIVE_COMPARATOR', 'label': 'Alendronate', 'description': "128 subjects participated in the study's Phase 2 (1-year double-blind, randomized, placebo-controlled, parallel group study).\n\n65 subjects were randomized to this arm. Oral alendronate dose: 5 mg/day, if body weight ≤25 kg; 10 mg/day, if body weight \\>25 kg.", 'interventionNames': ['Drug: Alendronate']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': "128 subjects participated in the study's Phase 2 (1-year double-blind, randomized, placebo-controlled, parallel group study).\n\n63 subjects were randomized to this arm. Oral placebo (inactive pills).", 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'Alendronate', 'type': 'DRUG', 'otherNames': ['Alendros'], 'description': 'As active drug, we used Alendros (Abiogen Pharma, Pisa, Italy), distributed to the patients in plain bottles and boxes (bearing only the center and patient codes).', 'armGroupLabels': ['Alendronate']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Placebo was distributed to the patients in plain bottles and boxes (bearing only the center and patient codes).', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Vatican City', 'country': 'Holy See', 'facility': 'CRR Fibrosi Cistica, Divisione Gastroenterologia, Ospedale Bambin Gesù', 'geoPoint': {'lat': 41.90268, 'lon': 12.45414}}, {'city': 'Grosseto', 'country': 'Italy', 'facility': 'CRR Fibrosi Cistica, Unità Operativa di Pediatria, Ospedale Misericordia', 'geoPoint': {'lat': 42.76296, 'lon': 11.10941}}, {'city': 'Messina', 'country': 'Italy', 'facility': 'CRR Fibrosi Cistica, Clinica Pediatrica, Policlinico Universitario di Messina', 'geoPoint': {'lat': 38.19394, 'lon': 15.55256}}, {'zip': '20145', 'city': 'Milan', 'country': 'Italy', 'facility': 'Istituto Auxologico Italiano IRCCS', 'geoPoint': {'lat': 42.78235, 'lon': 12.59836}}, {'city': 'Milan', 'country': 'Italy', 'facility': "Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Università degli Studi di Milano", 'geoPoint': {'lat': 42.78235, 'lon': 12.59836}}, {'city': 'Napoli', 'country': 'Italy', 'facility': 'CRR Fibrosi Cistica, Dipartimento Pediatria, Università Federico II', 'geoPoint': {'lat': 40.87618, 'lon': 14.5195}}, {'city': 'Orbassano', 'country': 'Italy', 'facility': 'CRR Fibrosi Cistica Adulti, Azienda Ospedaliera Universitaria San Luigi Gonzaga', 'geoPoint': {'lat': 45.00547, 'lon': 7.53813}}, {'city': 'Palermo', 'country': 'Italy', 'facility': 'CRR Fibrosi Cistica, Ospedale dei Bambini, ARNAS Civico', 'geoPoint': {'lat': 38.1166, 'lon': 13.3636}}, {'city': 'Roma', 'country': 'Italy', 'facility': 'CRR Fibrosi Cistica, Dipartimento di Pediatria, Policlinico Umberto I', 'geoPoint': {'lat': 44.99364, 'lon': 11.10642}}, {'city': 'Trieste', 'country': 'Italy', 'facility': 'CRR Fibrosi Cistica, Divisione di Pediatria, Istituto Burlo Garofolo', 'geoPoint': {'lat': 45.64953, 'lon': 13.77678}}, {'city': 'Verona', 'country': 'Italy', 'facility': 'CRR Fibrosi Cistica, Azienda Ospedalierouniversitaria di Verona', 'geoPoint': {'lat': 45.43854, 'lon': 10.9938}}], 'overallOfficials': [{'name': 'Maria Luisa Bianchi, M.D.', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Istituto Auxologico Italiano'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Istituto Auxologico Italiano', 'class': 'OTHER'}, 'collaborators': [{'name': 'Fondazione Telethon', 'class': 'OTHER'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'MD, First level manager', 'investigatorFullName': 'Maria Luisa Bianchi', 'investigatorAffiliation': 'Istituto Auxologico Italiano'}}}}